ReviewHuman gene therapy2020
Treating Cystic Fibrosis with mRNA and CRISPR.
Review in Human gene therapy, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 35 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
35 citing papers in PubMed.
- Finding the sweet spot between complexation and cytosolic release with different molecular weight branched polyethyleneimine polymers for mRNA delivery.Drug delivery and translational research · 2026Article
- Recent advances in the application of polymeric nanoparticles to the pulmonary delivery of mRNA.Nanomedicine (London, England) · 2026Review
- Transamniotic Fetal Delivery of Cystic Fibrosis (CF) Transmembrane Conductance Regulator (CFTR) mRNA.FASEB bioAdvances · 2026Article
- Extracellular vesicle-based delivery to airway basal cells for durable gene therapy in cystic fibrosis.Frontiers in bioengineering and biotechnology · 2026Review
- Robust peptide/RNA complexes prepared with microfluidic mixing for pulmonary delivery by nebulisation.Drug delivery and translational research · 2025Article
- Customizable Polymeric Nanoparticle Materials Optimized on Hypoxic Cells Facilitate mRNA Expression in the Lungs In Vivo.Advanced healthcare materials · 2025Article
- Advancing Therapeutic Strategies for Nonsense-Related Diseases: From Small Molecules to Nucleic Acid-Based Innovations.IUBMB life · 2025Review
- Revolutionizing Dentistry: Preclinical Insights and Future Applications of mRNA Vaccines in Dentistry-A Narrative Review.Dentistry journal · 2025Review
- Advancing the Battle against Cystic Fibrosis: Stem Cell and Gene Therapy Insights.Current medical science · 2024Review
- Review
- Combinatorial development of nebulized mRNA delivery formulations for the lungs.Nature nanotechnology · 2024Article
- Emerging Strategies for Immunotherapy of Solid Tumors Using Lipid-Based Nanoparticles.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2024Review
- Nanoparticle-Mediated Strategies for Enhanced Drug Penetration and Retention in the Airway Mucosa.Pharmaceutics · 2023Review
- Progress on RNA-based therapeutics for genetic diseases.Zhejiang da xue xue bao. Yi xue ban = Journal of Zhejiang University. Medical sciences · 2023Article
- Polymer nanoparticles deliver mRNA to the lung for mucosal vaccination.Science translational medicine · 2023Article
- Review
- Review
- Biodrug Delivery Systems: Do mRNA Lipid Nanoparticles Come of Age?International journal of molecular sciences · 2023Review
- mRNA in the Context of Protein Replacement Therapy.Pharmaceutics · 2023Review
- Features of CFTR mRNA and implications for therapeutics development.Frontiers in genetics · 2023Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors.
Funding
Abstract
Less than 20% of the protein coding genome is thought to be targetable using small molecules. mRNA therapies are not limited in the same way since in theory, they can silence or edit any gene by encoding CRISPR nucleases, or alternatively, produce any missing protein. Yet not all mRNA therapies are equally likely to succeed. Over the past several years, an increasing number of clinical trials with siRNA- and antisense oligonucleotide-based drugs have revealed three key concepts that will likely extend to mRNA therapies delivered by nonviral systems. First, scientists have come to understand that some genes make better targets for RNA therapies than others. Second, scientists have learned that the type and position of chemical modifications made to an RNA drug can alter its therapeutic window, toxicity, and bioavailability. Third, scientists have found that safe and targeted drug delivery vehicles are required to ferry mRNA therapies into diseased cells. In this study, we apply these learnings to cystic fibrosis (CF). We also describe lessons learned from a subset of CF gene therapies that have already been tested in patients. Finally, we highlight the scientific advances that are still required for nonviral mRNA- or CRISPR-based drugs to treat CF successfully in patients.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.